Affymax, Inc. and Takeda Pharmaceutical Company Limited announced the submission of a New Drug Application (NDA) to the US Food and Drug Administration (FDA) for the investigational agent peginesatide (formerly known as Hematide) for the treatment of anaemia associated with Chronic Renal Failure (CRF) in adult patients on dialysis.

Peginesatide is a synthetic, PEGylated peptidic compound that binds to and activates the erythropoietin receptor and thus acts as an Erythropoiesis Stimulating Agent (ESA). The agent is being co-developed by Affymax and Takeda. The NDA includes data from two phase III studies (Emerald 1 and 2) that evaluated the efficacy and safety of peginesatide, dosed once every four weeks, compared to epoetin alfa or epoetin beta, dosed more frequently (according to the product labels), in maintaining haemoglobin (Hb) levels. In the studies, CRF patients on dialysis who were receiving stable doses of epoetin, were randomized to receive once-monthly peginesatide or continue treatment with epoetin. The Emerald findings suggested that once-monthly peginesatide was similar to epoetin in maintaining Hb levels in CRF patients on dialysis with anaemia with a similar adverse event rate.

“The submission of the peginesatide NDA is an important milestone for Affymax and the result of dedication and diligence by both the Affymax and Takeda teams,” said John Orwin, chief executive officer, Affymax. “Anaemia is a debilitating condition for chronic renal failure patients on dialysis, and our program illustrates the potential of peginesatide as an important once-monthly therapeutic option for patients and providers, if approved. We look forward to working with the FDA as they review the data package for the peginesatide New Drug Application.”

“We're pleased to have partnered with Affymax on a submission that includes data from the largest phase III programme to support the initial registration of an ESA for the treatment of anaemia of chronic renal failure and the first to prospectively evaluate the cardiovascular safety of an ESA via an analysis of independently adjudicated cardiovascular events,” said Azmi Nabulsi, MD, M P H, president of Takeda Global Research & Development Centre, Inc. US. “To date, limited options for treating anemia in chronic renal failure patients on dialysis exist. The peginesatide New Drug Application represents an important step in the evaluation of a potentially new treatment for this common condition in chronic renal failure.”

Anaemia is a common complication in Chronic Kidney Disease (also referred to as CKD or CRF), because the impaired kidneys are not able to produce enough erythropoietin, the hormone that promotes the production of oxygen-carrying red blood cells. Research has shown that anaemia impacts the overall health and well being of CKD patients and is associated with increased rates of hospitalization and mortality. In severe or prolonged cases of anaemia, the lack of oxygen in the blood can cause serious and sometimes fatal damage to the heart and other organs.

ESAs, which stimulate red blood cell production, are commonly prescribed to treat anaemia in CRF. According to the Centres for Medicare and Medicaid Services, more than 95 percent of patients on dialysis in the US are currently receiving ESA treatment for anaemia in CRF.

Affymax and Takeda are collaborating on the development of peginesatide and plan to co-commercialize the product if approved in the United States. The product, upon approval, will be commercialized outside the United States by Takeda. The most common adverse events reported in the clinical studies were diarrhoea, cough, dyspnea, nausea, and muscle spasm.

Affymax, Inc. is a biopharmaceutical company committed to developing novel drugs to improve the treatment of serious and often life-threatening conditions.